title: Efficacy and safety of ipilimumab 3mg/kg in patients with pretreated, metastatic, mucosal melanoma.
abstract: BACKGROUND
  Mucosal melanoma is an extremely rare and aggressive malignancy that often remains undetected until it reaches an advanced stage, when effective treatment options are limited. The activity and safety of ipilimumab were assessed in an Expanded Access Programme (EAP) that included patients with metastatic, mucosal melanoma.
  
  
  METHODS
  Ipilimumab was available upon physician request for patients aged ⩾16years with stage III (unresectable) or IV skin, ocular or mucosal melanoma, who had failed or did not tolerate previous treatments and had no other therapeutic option available. Patients received ipilimumab 3mg/kg every 3weeks for four doses. Patients with stable disease or an objective response to ipilimumab were eligible for retreatment upon disease progression. Tumour assessments were conducted at baseline and week 12 using immune-related response criteria. Patients were monitored for adverse events (AEs), including immune-related AEs, within 3 to 4days of each scheduled visit.
  
  
  RESULTS
  Of 855 patients participating in the EAP in Italy, 71 (8%) had metastatic, mucosal melanoma. With a median follow-up of 21.8months, the response rate was 12% and the immune-related disease control rate was 36%. Median progression-free survival and overall survival were 4.3 and 6.4months, respectively. In total, 34% of patients reported treatment-related AEs of any grade, which were grade 3 or 4 in 9% of patients. AEs were generally manageable as per protocol-specific guidelines.
  
  
  CONCLUSIONS
  Ipilimumab may be a feasible treatment option in pretreated patients with metastatic mucosal melanoma, and warrants further investigation in prospective clinical trials.
authors.affiliation: Department of Medical Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy. Electronic address: michele.delvecchio@istitutotumori.mi.it.
authors.forename: Michele
authors.initials: M
authors.lastname: Del Vecchio
authors.affiliation: Department of Medical Oncology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy.
authors.forename: Lorenza
authors.initials: L
authors.lastname: Di Guardo
authors.affiliation: Melanoma, Cancer Immunotherapy and Innovative Therapy Unit, Istituto Nazionale Tumori Fondazione "G. Pascale", Naples, Italy.
authors.forename: Paolo A
authors.initials: PA
authors.lastname: Ascierto
authors.affiliation: Melanoma, Cancer Immunotherapy and Innovative Therapy Unit, Istituto Nazionale Tumori Fondazione "G. Pascale", Naples, Italy.
authors.forename: Antonio M
authors.initials: AM
authors.lastname: Grimaldi
authors.affiliation: Melanoma Cancer Unit, Veneto Institute of Oncology IOV-IRCCS, Padua, Italy.
authors.forename: Vanna Chiarion
authors.initials: VC
authors.lastname: Sileni
authors.affiliation: Melanoma Cancer Unit, Veneto Institute of Oncology IOV-IRCCS, Padua, Italy.
authors.forename: Jacopo
authors.initials: J
authors.lastname: Pigozzo
authors.affiliation: Department of Medical Oncology, Regina Elena National Cancer Institute, Rome, Italy.
authors.forename: Virginia
authors.initials: V
authors.lastname: Ferraresi
authors.affiliation: Department of Medical Oncology, Regina Elena National Cancer Institute, Rome, Italy.
authors.forename: Carmen
authors.initials: C
authors.lastname: Nuzzo
authors.affiliation: "Paolo Giaccone" Polyclinic University Hospital, Palermo, Italy.
authors.forename: Gaetana
authors.initials: G
authors.lastname: Rinaldi
authors.affiliation: Divisione Melanoma e Sarcomi Muscolo-Cutanei, Istituto Europeo di Oncologia IEO-IRCCS, Milan, Italy.
authors.forename: Alessandro
authors.initials: A
authors.lastname: Testori
authors.affiliation: Unità di Ricerca Traslazionale sul Melanoma, Divisione di Oncologia Medica del Melanoma, Istituto Europeo di Oncologia IEO-IRCCS, Milan, Italy.
authors.forename: Pier F
authors.initials: PF
authors.lastname: Ferrucci
authors.affiliation: Medical Oncology, Dermopathic Institute of the Immaculate IDI-IRCCS, Rome, Italy; Medical Oncology, Sant'Andrea Hospital, Sapienza University of Rome, Italy.
authors.forename: Paolo
authors.initials: P
authors.lastname: Marchetti
authors.affiliation: Medical Oncology, Dermopathic Institute of the Immaculate IDI-IRCCS, Rome, Italy.
authors.forename: Federica
authors.initials: F
authors.lastname: De Galitiis
authors.affiliation: San Martino Hospital, National Institute for Cancer Research, Genoa, Italy.
authors.forename: Paola
authors.initials: P
authors.lastname: Queirolo
authors.affiliation: San Martino Hospital, National Institute for Cancer Research, Genoa, Italy.
authors.forename: Elena
authors.initials: E
authors.lastname: Tornari
authors.affiliation: University Hospital Pisa, "Gathered Hospitals of Santa Chiara", Pisa, Italy.
authors.forename: Riccardo
authors.initials: R
authors.lastname: Marconcini
authors.affiliation: Department of Medical Oncology and Immunotherapy, University Hospital of Siena, Istituto Toscano Tumori, Siena, Italy.
authors.forename: Luana
authors.initials: L
authors.lastname: Calabrò
authors.affiliation: Department of Medical Oncology and Immunotherapy, University Hospital of Siena, Istituto Toscano Tumori, Siena, Italy.
authors.forename: Michele
authors.initials: M
authors.lastname: Maio
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000911:Antibodies, Monoclonal / Q000008:administration & dosage* / Q000009:adverse effects*; D018450:Disease Progression; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D000074324:Ipilimumab; D008297:Male; D008545:Melanoma / Q000188:drug therapy* / Q000473:pathology; D008875:Middle Aged; D009092:Mucous Membrane / Q000473:pathology*; D009362:Neoplasm Metastasis; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000911:Antibodies, Monoclonal; D000074324:Ipilimumab
keywords: Efficacy; Expanded access programme; Ipilimumab; Metastatic melanoma; Mucosal; Safety